The ability to modify the genome of an organism at a specifically targeted location is a long sought goal in the biological sciences. For example, such targeted modifications can repair genetic defects. Alternatively, genes (endogenous or heterologous) can be introduced into an organism at a pre-determined location. As yet another alternative, endogenous genes, or regulatory regions associated therewith, can be knocked out or altered. However, robust methods for such targeted genome modification have been challenging to develop.
The present invention provides a robust platform for modular assembly of customized and highly-effective nucleic acid editing tools. This platform enables rapid targeted modification of nucleic acids and genomes. The nucleic acid editing tools are based on an optimized Transcription Activator-like (TAL) effector endonuclease (TALEN) scaffold.